Core areas of focus

We apply innovative approaches, leading-edge science and world-class technical capabilities to our pursuit of medicines that improve the lives of patients. Our Research and Development efforts are primarily focused in a couple of areas in which we are proficient:

Strategic Principles

Pursue top quality science, internally and externally, balanced with medical need and commercial potential.

Excellence in research & development

Neurology research at Genocore Biotech is focused on identifying novel therapeutic approaches for devastating neurological and neurodegenerative conditions such as Alzheimer’s disease, Parkinson’s disease, amyotrophic lateral sclerosis and multiple sclerosis, aiming to move drug candidates into human clinical testing as safely, quickly and efficiently as possible.

Internally and with our many academic partners, we have a major focus on target discovery and validation using human stem cell models, functional cell-based screening, gene network and expression analysis, and study of disease-resistant neuronal populations.  We functionally validate candidate targets in the most relevant models and seek evidence of their involvement in human disease.

Subsequently, we explore with our colleagues across Genocore Biotech the therapeutic modality – small molecules, antisense oligonucleotides, antibodies or biologics – best suited to the patient population we are seeking to help. Our aim for each target is to create a complete package of information based on high-level mechanistic biology and rigorous testing that will give the program the best chance of success as it moves toward the clinic.

Immunology at Genocore Biotech is focused on exploring the immunobiology underlying autoimmune, inflammatory and fibrotic disease. Our goal is to discover and characterize molecules and pathways that can be targeted to develop biologic and small molecule therapeutics. Research in the department focuses on exploring the role of molecules, pathways and cell types in mediating pathological processes, developing novel target discovery models, elucidating mechanisms of disease and developing innovative biomarker strategies for disease and drug activity. Biology is explored from the molecular to the organismal level using state-of-the-art experimental approaches including proteomics, chemoproteomics and high-content systems approaches.

The hematology area at Genocore Biotech has grown out of our core hemophilia research group that discovered the recombinant clotting factor VIII and IX monomeric Fc fusion proteins and advanced them into clinical development for the improved treatment of hemophilia A and B, respectively. Moving beyond our initial assessment of these molecules, our scientists continue to advance our knowledge of their structure and activity relationships, as well as to better understand their mechanisms of action for half-life extension and generating tolerance/immunogenicity.


We measure success by our impact on the patients we serve.


Our group continues to explore novel therapies for the treatment of hemophilia, with an initial focus on further increasing the half-life of FVIII, and creating innovative therapies for people with hemophilia A and B who have developed neutralizing antibodies (inhibitors) against their current treatment options. We are now further expanding our research into related areas of nonmalignant hematology, such as sickle cell disease.

In all of our work, we seek to apply a combination of advanced techniques with pharmacology studies in relevant models to further understand the physiological interactions in these disease areas and better guide our protein engineering and drug development research. Our active collaborations with both academic and industry groups are essential. They enable us to apply the most advanced technologies and encompass the widest range of ideas to achieve our goals for transforming care of hematologic diseases with high unmet medical needs.


Medicinal Chemistry

The mission for the small molecule drug discovery team is to identify novel and differentiated oral small molecule drug candidates for the treatment of diseases of interest to Genocore Biotech and move these candidates into human clinical testing. We do this in close partnership with biologists, pharmacologists, toxicologists and others. This group utilizes high-throughput and phenotypic screening to discover chemical leads. The leads are optimized for potency, selectivity, cellular activity and ADME/PK properties via iterative cycles of structure-based drug design, hypothesis-driven approach and parallel organic synthesis. We work with preclinical imaging and chemoproteomics groups in designing probes to evaluate the target engagement of our clinical candidates. Genocore Biotech's growing small-molecule drug-discovery group will enhance the pipeline by expanding the drug target pool and pharmacologic treatment modes accessible to protein therapeutics.